Ascendis Pharma A/S announced on the 28th February 2019, on the rare disease day that the U.S. Food and Drug Administration, FDA, has granted Orphan Drug Designation (ODD) to TransCon CNP.

The Sunnaas Rehabilitation Hospital is conducting an observational study on the prevalence of medical complications in adults with achondroplasia and their impact on daily activities entitled "The Norwegian Adult Achondroplasia Study".

Observational Study Investigating Clinical & Anthropometric Characteristics of Children With Achondroplasia, conducted by Therachon, is an observational study to investigate natural history of achondroplasia. It is currently recruiting for a number of countries.

LIAISE is the Lifetime Impact of Achondroplasia Study in Europe. It will gather information on the clinical and medical interventions of people with achondroplasia, individuals' and caregivers' quality of life and the impact of this condition on education, employment and social life.

On the 28th Nov 2018, Ascendis Pharma A/S announced positive preliminary results from a phase 1 trial of TransCon CNP.

Here you will find the highlights of the World Orphan Drug Congress, WODC 2018, where pharmaceutical companies, contract research organizations (CROs) and Health Technology Assessment agencies were heavily represented, with the participation of some patient organizations, discussing pathways related to orphan drug development and clinical trials. The active participation of patients in research was also a key topic.